Clinical Research Bridges New Ideas to Advances in Care

On the journey to better treatments, clinical research studies are the bridge between new ideas and proven advances in care.

“Mommy, my mouth feels funny,” Kaylah Talamantez repeatedly told her mom during the winter she was 4 and couldn’t shake what seemed like a bad cold or maybe persistent allergies. Her doctors told the family to give the medicine some time to take effect.

But the weeks dragged into months, and mom Kristina Talamantez finally had enough. A visit to an ear, nose and throat specialist in April resulted in an MRI. The next day the family was on their way from their Stanwood, Washington, home to Seattle Children’s where Kaylah was diagnosed with rhabdomyosarcoma (RMS), the most common soft-tissue cancer in children. In the span of a few months, the quickly growing tumor behind Kaylah’s nose and close to her brain was already reaching through her palate and starting to push the little girl’s eyes further apart.

Cancer specialist Dr. Doug Hawkins met with the family to explain the options for Kaylah’s treatment – including a clinical research study he was leading to test if adding a new medication to the standard treatment would improve a child’s chances of being cured.

Testing the benefits

Clinical research studies allow doctors and researchers to evaluate new ways to detect, control and treat medical conditions by systematically testing the risk-to-benefit ratio of a new approach compared to the current treatment. Participation in these carefully planned studies is completely voluntary.

“These studies are the link between ideas or new observations in the laboratory and the reality of being able to offer better options to patients. That’s why they are so crucial,” says Dr. Bonnie Ramsey, a pioneer in cystic fibrosis (CF) treatment and research whose work has resulted in life-enhancing improvements for people with CF.

Any research that involves humans – whether it is their personal health information, samples of tissue, or active participation in trying a new treatment – is considered clinical research.

Participating in progress

“Dr. Hawkins told us that current treatments were based on past research studies that showed what worked best,” recalls Talamantez. “We wanted to be part of finding better ways to get kids healthy so we decided to join the study. I knew that if anything went awry or wasn’t working, Kaylah’s doctors would step in and say ‘let’s try something else’.”

Kaylah joined a five-year study that involved patients at nearly 200 medical centers around the world. The study compared the standard chemotherapy treatment for RMS to the standard treatment plus an additional medicine called irinotecan, a type of chemotherapy that works by blocking an enzyme in cells that prevents them from dividing properly. The study was designed to see if the new treatment would improve the cure rate of about 66%. Hawkins and others had been studying how to use irinotecan in RMS for nearly 15 years and were hopeful since both the laboratory studies and early studies with people showed promise.

“Clinical research studies are the link between new observations in the laboratory and the reality of offering better options to patients. That’s why they are so crucial.”

– Dr. Bonnie Ramsey

Fewer harsh side effects

The new treatment was so successful it resulted in a call for a new standard of care for RMS – though not exactly for the reason doctors initially hoped. Though the overall cure rate remained the same, the new treatment caused significantly fewer harsh side effects like life-threatening infections and the need for blood transfusions.

“We were disappointed we weren’t able to improve the survival rate,” acknowledges Hawkins, “but very excited to identify a way to make treatment more tolerable for patients and improve their quality of life.”

Kaylah, now 10 and healthy, recently celebrated her fifth anniversary out of treatment and doesn’t remember much about the time that she was sick. “She didn’t realize that getting all those treatments wasn’t normal for a 4-year-old. There were lots of other kids at the hospital doing the same thing she was,” says her mom.

“Each new study builds on learnings from prior studies. The gains are step-by-step, but over time the improvement has been tremendous,” says Hawkins. “Unfortunately, we still don’t have good answers for too many kids. Until we can cure every child with no side effects, we still have work to do.”

Researchers at Seattle Children’s are studying a wide range of pediatric health issues. If you want to know more about research underway in an area of interest to you, please call Brenda Majercin at 206-987-2153 or email her.

Families and patients can browse Seattle Children’s currently-enrolling clinical research studies on the Research Studies and Clinical Trials Web Hub. The hub also includes information about what clinical research is and why patient families may want to consider participating.

Published in Connection magazine, Winter 2016